In this second report on MDA's Clinical Conference, updates on experimental therapies in development for six neuromuscular diseases are described
The progress of several experimental therapies currently in development for neuromuscular diseases was discussed at MDA's 2012 Clinical Conference, held in Las Vegas March 4-7.
Quest takes a look at gene therapy, 'antisense' and other cutting-edge scientific approaches and how they're being applied to diseases in MDA's program
Antisense oligonucleotides block flawed genetic instructions
Antisense oligonucleotides — also called antisense, oligos, or simply AONs — are pieces of genetic code that keep other genetic code from being processed. Designed to pair up with a particular sequence of DNA or RNA, AONs can change, block or destroy targeted genetic instructions in a variety of ways.
New treatments bring relief in inflammatory muscle disease, but patience is required
Giovanna Albers, 38, has recovered from her polymyositis and no longer takes medication for it. Her weight is back to normal, and she "lives and breathes" in the water. Photo by Tim Parker